Phase III Study of Induction and Consolidation Chemotherapy With Venetoclax in Patients With Newly Diagnosed AML or MDS-EB-2
This research study is looking for adults aged between 18 and 75 who have been recently diagnosed with Acute Myeloid Leukaemia (AML) or a specific type of Myelodysplastic Syndrome (MDS-EB-2). It aims to find a better way to treat these conditions. Patients will receive standard chemotherapy, but some will also get a new drug called venetoclax, while others will get a placebo (a dummy drug). The study wants to see if adding venetoclax improves treatment results. Participants will be randomly assigned to either the venetoclax or placebo group, and researchers will compare how well each group responds. This is a "Phase III" study, which means it's one of the final steps before a new treatment might become widely available.
At a glance
What is this study about?
This study is for people who have recently found out they have Acute Myeloid Leukaemia (AML) or a specific type of Myelodysplastic Syndrome called MDS-EB-2. These are cancers that affect the blood and bone marrow, which is the spongy material inside your bones where blood cells are made. The study is trying to find out if adding a drug called venetoclax to the usual chemotherapy treatment can improve how well people respond.
Imagine you have a team of doctors trying to fight a fire. Standard chemotherapy is like a powerful fire hose. This study is testing if adding venetoclax is like bringing in a second, specialised tool that might help put out the fire more effectively. Half the patients in the study will get the usual chemotherapy plus venetoclax, and the other half will get the usual chemotherapy plus a dummy drug (placebo). This is to fairly compare if venetoclax makes a difference.
The study starts with an initial phase to figure out the best dose of venetoclax. After that, eligible patients will be randomly assigned to one of the two treatment groups. Doctors will then closely watch how each group responds to the treatment during initial (induction) and follow-up (consolidation) phases. For some, a stem cell transplant might be considered later, depending on their individual situation.
Key takeaways
- This study is for newly diagnosed adults (18-75) with AML or MDS-EB-2.
- It tests a new drug (venetoclax) with standard chemotherapy versus chemotherapy alone.
- It's a Phase III study, aiming to find better treatment options.
- Participation involves chemotherapy, possibly venetoclax or a placebo, and regular monitoring.
- You will be randomly assigned to a treatment group, and neither you nor your doctor will know which one.
Who may be eligible?
To join this study, you generally need to be an adult between 18 and 75 years old who has been newly diagnosed with Acute Myeloid Leukaemia (AML) or a specific type of Myelodysplastic Syndrome (MDS-EB-2). Your doctors must believe you are strong enough to receive the usual intensive chemotherapy treatment.
You also need to meet certain health requirements. Your kidneys and liver need to be working well, and your general health and how active you are will be assessed. You shouldn't have had previous chemotherapy for your AML, although some initial short-term medication for high white blood cell counts is allowed. If you've had certain treatments for MDS before, you might still be eligible, but these medications need to be stopped a few weeks before starting the study.
There are also some requirements for women who could get pregnant. They must either be past menopause or have had certain surgical procedures that prevent pregnancy. You also shouldn't be taking certain types of medication that can interfere with the study drug.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 18 and 75 years old?
- Have you been newly diagnosed with AML or MDS-EB-2?
- Are you generally well enough for intensive chemotherapy?
- Do you have good kidney and liver function?
- Have you not received prior chemotherapy for AML (except certain short-term medications)?
- If you are a woman, are you past menopause or have you had certain surgeries to prevent pregnancy?
What does participation involve?
If you join this study, you'll first go through some checks to make sure it's suitable for you. This includes blood tests and other examinations. There's an initial period where doctors work out the best dose of the study drug.
Once that's done, if you continue, you'll be randomly assigned to either receive the study drug (venetoclax) or a dummy drug (placebo), both given alongside your standard chemotherapy. This initial treatment, called 'induction', usually involves two cycles. If your cancer responds well, you'll then move into a 'consolidation' phase, which is further treatment to help keep the cancer away. This could involve more chemotherapy, and for some, doctors might recommend a stem cell transplant.
Throughout the study, you'll have regular hospital visits for blood tests, assessments, and to receive your treatment. Doctors will monitor how you're responding and check for any side effects. The total duration of your participation will depend on how you respond to treatment and what follow-up care is needed, but it will involve multiple cycles of treatment and ongoing monitoring.
Potential risks and benefits
Locations (91)
- Tirol Kliniken GmbHVerified postcodeInnsbruck, Austria· Not yet recruiting
- Kepler Universitaetsklinikum GmbHVerified postcodeLinz, Austria· Not yet recruiting
- Ordensklinikum Linz GmbHVerified postcodeLinz, Austria· Not yet recruiting
- Landeskrankenhaus (LKH) Rankweil, Interne E am Landeskrankenhaus RankweilVerified postcodeRankweil, Austria· Not yet recruiting
- Gemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbHVerified postcodeSalzburg, Austria· Not yet recruiting
- Hanusch Krankenhaus Der Wiener GebietskrankenkasseVerified postcodeVienna, Austria· Not yet recruiting
- Ziekenhuis Aan De StroomVerified postcodeAntwerp, Belgium· Not yet recruiting
- Az St-Jan Brugge-Oostende A.V.Verified postcodeBruges, Belgium· Not yet recruiting
- Universitair Ziekenhuis BrusselVerified postcodeBrussels, Belgium· Recruiting
- Katholieke Universiteit te LeuvenVerified postcodeLeuven, Belgium· Not yet recruiting
- Algemeen Ziekenhuis DeltaVerified postcodeRoeselare, Belgium· Recruiting
- CHU UCL NAMUR - Mont GodinneVerified postcodeYvoir, Belgium· Recruiting
Common questions
What is Acute Myeloid Leukaemia (AML)?
AML is a type of cancer that starts in the bone marrow and affects the cells that would normally develop into different types of blood cells. It's a fast-growing cancer.
What is a 'placebo'?
A placebo is an inactive substance, sometimes called a 'dummy drug', that looks just like the real medicine. It's used in studies to fairly compare the new drug's effects.
Why is this called a 'Phase III' study?
Phase III studies are typically the last stage of testing a new treatment before it's considered for wide use. They involve many people and compare the new treatment with existing ones to confirm safety and effectiveness.
Will I know if I'm getting venetoclax or the placebo?
No, this is a 'double-blind' study. This means neither you nor your study doctors will know whether you are receiving venetoclax or the placebo. This helps ensure the study results are fair and unbiased.
Can I stop participating in the study at any time?
Yes, absolutely. Your participation is completely voluntary, and you can withdraw from the study at any point without it affecting your future medical care.
How to find out more
Hartmut Doehner, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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